Understanding why renal tumors in TSC resist therapy to guide new nano-based treatments
Official title: Elucidation of Tumor Resistance Mechanisms in Tuberous Sclerosis Complex-Associated Renal Angiomyolipoma for the Design of Novel Nanotherapies
Funded at Brigham and Women's Hospital · NIH-11345874 · Data updated 2026-11-12
Researchers are studying why kidney tumors in TSC patients resist current drugs, with the goal of designing smarter, more effective treatments that could help people with TSC-related AML.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brigham and Women's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | June 1, 2023 |
| Project end | May 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $394,013 |
| NIH project number | 5R01CA269583-04 |
| Project ID | NIH-11345874 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with Tuberous Sclerosis Complex who develop renal angiomyolipoma or who have genetic or cellular samples related to TSC AML.
✕Less likely to help
People without TSC-related AML or without access to related tissue samples may not benefit.
What this research studies
This project uses patient-derived iPSCs to create kidney tumor organoids that mimic real tumors. The team will study resistance mechanisms at the cellular and molecular levels to identify new therapeutic targets. They will test novel nanotherapies in these organoids and in animal models to see if they can better shrink or control tumors while reducing side effects. The work aims to translate laboratory findings into improved options for patients who struggle with AML due to TSC.
Why it matters
Potential benefit: If successful, the research could lead to more effective, long-lasting treatments for TSC-associated AML with fewer side effects.
How similar studies have gone: Previous work shows that AML organoids derived from TSC2-/- iPSCs can model human AML features, supporting the potential for this approach to yield actionable insights.
Where this research is happening
-
Brigham and Women's Hospital
Boston, United States Active
Researchers
- Principal investigator: Lemos, Dario — Brigham and Women's Hospital
- Study coordinator: Lemos, Dario
Related research
Other NIH-funded projects on the same topics.
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VIP Interneurons and Brain Circuits in Tuberous Sclerosis
A study to understand how loss of a brain gene (Tsc1) affects VIP interneurons and the frontal brain region in people and models with Tuberous Sclerosis Complex.
-
Clinical Readiness to Treat Tuberous Sclerosis–Related Neuropsychiatric Issues
A large NIH grant aims to ready treatments for the neuropsychiatric and developmental challenges that many people with tuberous sclerosis complex (TSC) face, including autism-like symptoms and sleep or sensory problems.
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Immune causes of kidney disease in tuberous sclerosis
The team is looking at whether immune cells and a protein called B7-H3 help kidney cysts and growths in people with tuberous sclerosis complex.
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Improving care for autism, learning, sleep, and behavior issues in tuberous sclerosis
This project develops user-friendly tools, follows patients over time, and searches for sleep, sensory, and brain-activity markers to help people with tuberous sclerosis complex who have autism, intellectual disability, or other neuropsychiatric challenges.
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Investigating the role of specific proteins in kidney disease related to Tuberous Sclerosis Complex.
This study is looking at how certain proteins affect kidney problems in people with Tuberous Sclerosis Complex, with the hope of finding better treatments that could help improve kidney health and reduce the need for long-term medication.
-
Understanding early communication in children with tuberous sclerosis complex and its link to autism.
This study is looking at how babies with tuberous sclerosis complex learn to communicate and how those skills might relate to the severity of autism, by watching videos of their vocal sounds and interactions over time.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.